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2/13/2012

Odd Couplings – Story by Rick Mullins, Chemical Engineering & News – Volume 90 Issue 7 | February 13, 2012

Drug Firms Engage in Nontraditional Research Partnerships in a Bid to Get Closer to the Patient In the pantheon of companies competing in the health care market, Proteus Biomedical is unique. A start-up based in Redwood City, Calif., Proteus has developed technology to attach a biodegradable digital device to a pill. After a patient swallows [...]

Roche, PTC & SMA Foundation Agreement to Develop a Treatment for SMA Receives Extensive Coverage

On November 29, 2011, Roche, PTC Therapeutics and the SMA Foundation announced the signing of an agreement to develop a treatment for Spinal Muscular Atrophy. Several media sources reported on the agreement including BioCentury, BioWorld Today, Bloomberg Businessweek, Chemical & Engineering News, Clinical Professionals, Dow Jones VentureWire, Drug Discovery News, FierceBiotech, Genetic Engineering and Biology News, [...]

ProteinTech Interview with The Spinal Muscular Atrophy Foundation

Spinal Muscular Atrophy (SMA) is a genetic neuromuscular disease characterized by motor neuron loss and muscle atrophy and weakness. The disease is caused by deletion or mutational inactivation of the SMN1 gene that encodes survival of motor neuron (SMN) protein, critical to motor neuron health and survival. The disease typically presents in early infancy and [...]

Biogen Idec and Isis Pharmaceuticals Announce Global Collaboration for Antisense Program Targeting Spinal Muscular Atrophy

Biogen Idec and Isis Pharmaceuticals Announce Global Collaboration for Antisense Program Targeting Spinal Muscular Atrophy – Biogen Idec has Option to Develop and Commercialize Promising Compound for Most Common Genetic Cause of Infant Mortality – – Biogen Idec’s Expertise in Neurology to Aid in Rapid Development of ISIS-SMNRx – SMA is a genetic neuromuscular disease characterized [...]

Biogen May Spend as Much as $299 Million on Isis Treatment for Children

Biogen May Spend as Much as $299 Million on Isis Treatment for Children Bloomberg News by Robert Langreth January 4, 2012 Biogen Idec Inc. (BIIB) will gain Isis Pharmaceuticals Inc. (ISIS)’s drug for an inherited muscle-weakening disease that afflicts children, in a development deal that may cost as much as $299 million. Biogen, the world’s biggest maker [...]

Salvar a Arya

El País by Sandro Pozzi December 26, 2011 Las cifras ayudan a entender la batalla que libran Dinakar Singh y Loren Eng, millonario y exsocio del banco Goldman Sachs, y su esposa, Loren Eng, por su hija Arya. Entre los más de 312 millones de habitantes de EE UU, solo 9.000 tiene la enfermedad infantil [...]

Isis Initiates Phase 1 Clinical Study of ISIS-SMNRx in Patients with Spinal Muscular Atrophy

CARLSBAD, Calif., December 19, 2011 – Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it has initiated a Phase 1 study of ISIS-SMNRx in patients with spinal muscular atrophy (SMA). SMA is a severe motor-neuron disease that is the leading genetic cause of infant mortality. Isis is developing ISIS-SMNRx as a potential treatment for all [...]

Roche Signs Agreement with PTC Therapeutics to Advance Treatment for Spinal Muscular Atrophy (SMA)

Collaboration offers new hope for a potential treatment for the leading genetic cause of death in infants and toddlers Basel, Switzerland, South Plainfield, NJ, USA and New York, NY, USA – November 29, 2011 – Roche (SIX: RO, ROG; OTCQX: RHHBY), PTC Therapeutics, Inc. (PTC) and the SMA Foundation, announced today a licensing agreement for [...]

A Thousand More

A family is determined to give their disabled son a whole and vital life. In the midst of a great burden, one small child – with a seemingly endless supply of love – is the blessing that holds a family together. See the project at http://mediastorm.com/training/a-thousand-more

Bloomberg Markets: “To Save a Child”

Video – September 7, 2011 For the full story, please see the October edition of Bloomberg Markets.

FSMA Announces Major Breakthrough in Development of Drug Candidate for Currently Untreatable Disease

June 22, 2007, Libertyville, IL – Families of Spinal Muscular Atrophy (FSMA) is pleased to announce the selection of a Clinical Candidate for Spinal Muscular Atrophy through its program being conducted at deCODE chemistry. At the same time FSMA is now extending its contract with deCODE to continue work towards an Investigational New Drug (IND) [...]

NIH/NINDS Director Highlights Remarkable Scientific Advances in SMA Research in Congressional Hearing

Washington, D.C. – March 29, 2007 - In Senate Subcommittee on Labor-HHS-Education Appropriations hearings this week, National Institute of Neurological Disorders and Stroke Director, Story C. Landis Ph.D., testified on the remarkable scientific advances that have been made in spinal muscular atrophy research over the past decade. This is an excerpt from her remarks: A decade [...]

Treatment Extends Survival in SMA Mouse – Researchers Optimistic About HDAC Inhibitors

story by Robinson, Richard Spinal muscular atrophy (SMA) — a juvenile form of motor neuron disease leading to weakness and wasting of voluntary muscles — is caused by a loss of the SMN1 gene, but even the most severely affected patients retain a functional copy of an almost identical gene, SMN2. In mice, increasing SMN2 [...]

Treatment Extends Survival in Mouse Model of Spinal Muscular Atrophy.

Drug therapy can extend survival and improve movement in a mouse model of spinal muscular atrophy (SMA), new research shows. The study, carried out at the NIH’s National Institute of Neurological Disorders and Stroke (NINDS), suggests that similar drugs might one day be useful for treating human SMA. “This study shows that treatment can be [...]

Spinal Muscular Atrophy Foundation Receives CNF Advocacy Award of Merit

New York, NY – November 10, 2006 – The Spinal Muscular Atrophy (SMA) Foundation announced today that it has received the 2006 Advocacy Award of Merit from the Child Neurology Foundation (CNF). The award was presented to the SMA Foundation during the Annual Meeting of the Child Neurology Society in Pittsburgh on October 20. Founded [...]

Lexicon Genetics Awarded Additional Grant for Study of SMA

The Woodlands, Texas – October 11, 2006 – Lexicon Genetics Incorporated (Nasdaq: LEXG) announced today that its research program to identify targets that may be important in the development of drugs to prevent or treat spinal muscular atrophy (SMA) has been extended for an additional year by the United States Army Medical Research & Materiel [...]

SMA Foundation Announces Collaboration with PTC Therapeutics

New York, NY – June 7, 2006 – The Spinal Muscular Atrophy Foundation today announced that they have initiated a research and development collaboration with PTC Therapeutics, Inc. (PTC). The collaboration is designed to leverage PTC’s proprietary Gene Expression Modulation by Smallmolecules (GEMS) technology to identify and develop new small molecule therapeutics for use in [...]

New Push for NIH Funding Urged

By Jonathan D. Rockoff Sun Reporter WASHINGTON, D.C.— May 20, 2006 – Senators urged research advocacy groups and universities yesterday to step up efforts to win more federal funding for the National Institutes of Health, the federal agency that funds most of the country’s medical research but is facing a third consecutive year of cuts. [...]

The Today Show, December 26, 2005, Featuring the SMA Foundation

On Monday, December 26, 2005, NBC’s The Today Show featured one family’s fight against thgenetic motor neuron disease Spinal Muscular Atrophy and the impact SMA has on parensearching for a treatment for their child. The show highlighted the daily struggle that 6-year-old Arya faces battling the effects of SMA and how she and her parents, [...]

New Motor Neuron Center Will Tackle Neurodegenerative Diseases

The birth of any organization dedicated to reducing human suffering is fueled by hope and propelled by the desire to achieve extraordinary goals; this was certainly true on Nov. 2 when Columbia’s Motor Neuron Center was launched. The gathering of about 40 leading researchers and clinicians from different disciplines at CUMC and Morningside, committed to [...]

Drug Provides Glimmer of Hope for Children Suffering Fatal Genetic Disease

Stanford University News by Krista Conger Ching Wang, MD, PhD, didn’t sign up for his pediatric neurology residency in 1990 to watch children die. But, as in nearly any medical specialty, there are some fatal diseases for which no effective treatment exists. Frustrated after delivering grim news to one too many sets of parents, Wang [...]

SMA Foundation Makes the Case for Research in Rare Disease

By Debbie Strickland The errant gene has been isolated, the National Institutes of Health has made the disease a priority and now a young foundation is pitching biotech companies to develop a cure for spinal muscular atrophy, a disabling, often fatal genetic disorder that afflicts some 25,000 Americans. Dinakar Singh and Loren Eng launched the [...]

SMA: Race Is On to Conquer Devastating Childhood Disease

SMA: Race is on to Conquer Devastating Childhood Disease When a young child is slow to meet developmental milestones such as rolling over, crawling, taking his first steps – or perhaps reaches them and then slows or slides back in development – parents grow concerned and look for explanations. For about 1 out of every [...]

Forbes: For Arya

-by Robert Langreth A Wall Street Power Couple Race to Find a Cure for Their Daughter Before It Is Too Late Arya Singh was born in March 2000, sat up and crawled on schedule but never really walked right. Pediatricians said at first that she was simply a late bloomer. But at 17 months she [...]

Deadly Disorder Kills Kids, But Lacks Funding

NEW YORK — March 7, 2005 – Two-year-old Owen Cain cannot lift his head or move his limbs. He’s fed through a tube, and even the simple act of sitting in a chair can trigger an alarming breathing spasm. The child has Spinal Muscular Atrophy, or SMA – the leading genetic killer of infants and [...]

Neurology Today: “Advocacy for Neurological Disease: The ‘Family Business’ No Family Wants”

By Gina Shaw They don’t have MD or PhD degrees. They don’t run research laboratories, direct hospitals, or oversee major academic medical centers. Yet they have helped changed the course of neurological research, driving tens of millions of research dollars – both private and federal – into the quest for treatments and cures for diseases [...]

The New York Times: “A Deadly Disease of Infants Attracts New Research Money,” by Anahad O’Connor

When Ellen Goldstein of Brooklyn gave birth last November to her only child, Owen, medical tests offered no clues that five months later he would be crippled by a deadly and irreversible genetic disease. Tests shortly before Owen was born revealed no abnormalities, and a physical evaluation right after his birth showed he was in [...]

North County Times: “Cure for Genetic Disease Requires Money,” by George Eng

Fifty years ago today, James Watson’s and Francis Crick’s scientific paper ignited a revolution in modern science. Fidgeting with cardboard cutouts and X-ray photographs, Watson and Crick discovered the spiral staircase structure of DNA. Their description of the double-helix sparked a torrent of research into DNA that today has us on the verge of finding [...]

Pittsburgh Post Gazette: “Funding Needed to Cure a Disease that Makes Children Waste Away”

By Loren A. Eng Fifty years ago, Francis Crick and James Watson ignited a revolution in modern science. Toying with a collection of cardboard cutouts and X-ray photographs, the two scientists discovered the structure of deoxyribonucleic acid, known as DNA. Their spontaneous observation of the “double helix” sparked a torrent of research into DNA that [...]

Parents Magazine: Parents Support Circle, “Arya, 3, Has Spinal Muscular Atrophy”

Nightline: UpClose with Ted Koppel. “Arya Singh.”

ABC NEWS UPCLOSE: ARYA SINGH ANCHORED BY: TED KOPPEL WITH GUESTS: LOREN ENG AND DINAKAR SINGH JANUARY 21, 2003 TED KOPPEL, ABC NEWS (OC) “Up Close” tonight, a young girl, a two-year-old, whose name is Arya. She lives in New York City, with her parents and baby brother. Arya has a genetic disease that the [...]

The New York Times: “When Not Knowing a Celebrity Could Prove Fatal”, by Adam Cohen

By ADAM COHEN DECEMBER 29, 2002 When Loren Eng, a briskly efficient Stanford Business School graduate, lobbies Congress for research funds for the disease that is crippling her 2-year-old daughter, she takes along almost everything she needs to make an airtight case. She has figures showing that the disease, spinal muscular atrophy, is badly underfinanced [...]

WABC-TV, New York: “Family Helping Others Learn More About SMA.”

New York, WABC, September 19, 2002 — For parents, few things are more difficult than watching their child struggle with a serious illness. When a muscle disease threatened their daughter, a frustrated family looked for a treatment and then found a way to help others facing the same crisis. Now, ABC7′s On Call with Dr. [...]

Columbia University Press: “Columbia University & Columbia-Presbyterian Med Ctr Open New SMA Clinic”

WHEN & WHERE: Tuesday, September 17, Noon Harkness Pavilion 180 Fort Washington Avenue New York NY WHAT: The Departments of Neurology Columbia University and at Columbia Presbyterian Medical Center are celebrating the opening of the SMA (spinal muscular atrophy) Clinic, a center devoted to the treatment and management of a group of diseases that affect [...]